Canakinumab in paediatric and young adult sickle cell anaemia patients
Research type
Research Study
Full title
A multiple-dose, subject- and investigator-blinded, placebo-controlled, parallel design study to assess the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia
IRAS ID
213752
Contact name
David Rees
Contact email
Sponsor organisation
Novartis Pharma Services AG
Eudract number
2016-002101-19
Duration of Study in the UK
1 years, 6 months, 18 days
Research summary
This study is in patients (12-20 years old) with sickle cell anaemia (SCA). This inherited disease affects ~312,000 people born each year and causes abnormally shaped red blood cells which results in inflammation, chronic pain, fatigue and low red blood cell levels with acute episodes of severe pain. Patients experience a reduced quality of life with severe organ damage and early mortality.
Current limited treatment options include hydration, pain relief and antibiotics to manage the acute episodes of pain (crises). Hydroxyurea is the only FDA-approved therapy for crises, working by increasing the ratio of normal:mutated red blood cells but has uncertain side effects. Frequent blood transfusions are used and bone marrow transplants are effective but also come with long term side effects.
The treatment being investigated in this study, canakinumab, is a marketed drug (Ilaris), approved for the treatment of a group of conditions known as cryopyrin-associated periodic syndromes (CAPS), systemic juvenile idiopathic arthritis (sJIA) and acute gouty arthritis attacks. Ilaris is a monoclonal antibody which blocks the action of a group of cells responsible for blood vessel inflammation and reduces their obstruction by abnormal red blood cells, thus reducing pain and organ damage.
This study is subject- and investigator-blinded which means that participants and the doctors/nurses treating them will not know which treatment they have been randomly assigned (canakinumab or a placebo).
This is a parallel study which means that patients receiving canakinumab or placebo will do so at roughly the same time. Patients will have 6 blinded monthly subcutaneous injections, then an optional open-label 6 month treatment period.
The study will investigate how canakinumab works to reduce average daily pain of SCA patients, recorded in an electronic diary.
Patients will be on study for approximately 13 months and the aim is to recruit 16-20 patients in 1-2 UK sites.REC name
London - Central Research Ethics Committee
REC reference
16/LO/1874
Date of REC Opinion
22 Nov 2016
REC opinion
Further Information Favourable Opinion